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Rhythm Pharmaceuticals Announces FDA Approval of IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity

First and only FDA-approved therapy for the treatment of acquired hypothalamic obesity, a rare disease characterized by accelerated and sustained weight gain caused by hypothalamic injury or dysfunction

Rhythm Pharmaceuticals, Inc. (Nasdaq: RYTM), a global commercial-stage biopharmaceutical company focused on transforming the lives of patients living with rare neuroendocrine diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for IMCIVREE® (setmelanotide) to treat patients living with acquired hypothalamic obesity (HO).

Acquired HO is a rare disease characterized by accelerated and sustained weight gain caused by an injury to the hypothalamus or hypothalamic dysfunction. With this label expansion, IMCIVREE is indicated to reduce excess body weight and maintain reduction long term in adults and pediatric patients aged 4 years and older with acquired HO.

“IMCIVREE is now the first and only FDA-approved therapy for acquired HO, offering a targeted approach that addresses the underlying biology of this disease and meets a critical unmet need for patients who previously had no treatment options,” said David Meeker, M.D., Chairman, Chief Executive Officer and President of Rhythm. “This is a transformative milestone for Rhythm and reinforces our commitment to bringing meaningful therapies to patients living with rare MC4R pathway diseases.”

The MC4R pathway is responsible for controlling physiological functions such as energy expenditure, hunger, and weight regulation. Acquired HO most frequently follows tumors and their treatment or other hypothalamic injury or dysfunction. Based on analysis of the literature, tumor registries and claims data, Rhythm estimates there are approximately 10,000 people living with acquired HO in the U.S.

“Having a therapy for individuals and families affected by acquired hypothalamic obesity has the potential to be transformational,” said Amy Wood, Executive Director and Founder of the Raymond A. Wood Foundation. “We’ve seen firsthand the devastating impact acquired hypothalamic obesity has on patients’ and families’ lives, including relentless hunger and accelerated and sustained weight gain. IMCIVREE offers hope and a path forward for thousands of patients who have long been without options.”

The approval is supported by the positive pivotal Phase 3 TRANSCEND trial of setmelanotide in 142 patients with acquired HO. The global study met its primary endpoint, with a statistically significant -18.4% placebo-adjusted reduction in body mass index (BMI). For the primary endpoint of mean BMI change from baseline, study participants on setmelanotide therapy (n=94) achieved a -15.8% reduction compared with a +2.6% increase among patients on placebo (n=48) at 52 weeks (p<0.0001). Setmelanotide was generally well tolerated in the Phase 3 trial. The most common adverse events (affecting >20% of participants) were skin hyperpigmentation, nausea, vomiting and headache.

“Setmelanotide has shown effectiveness in targeting the underlying biology of acquired HO,” said Ashley Shoemaker, M.D., MSCI, Associate Professor of Pediatrics, Pediatric Endocrinology at Vanderbilt Health. “Patients treated with setmelanotide experienced meaningful reductions in BMI and hunger, demonstrating the therapy’s ability to deliver clinically significant outcomes in both children and adult patients. Acquired HO is a severe disease that requires early and proactive management. With the availability of IMCIVREE, physicians can offer a targeted therapy.”

IMCIVREE® (setmelanotide) is also approved in the U.S. and Europe in adult and pediatric patients aged 2 years and older with syndromic or monogenic obesity due to Bardet-Biedl syndrome (BBS) or Pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin type 1 (PCSK1), or leptin receptor (LEPR) deficiency.

Rhythm is committed to supporting access for patients to its medicines and IMCIVREE® (setmelanotide) will be available for patients in the U.S. immediately. Rhythm InTune provides personalized, ongoing educational support for individuals living with certain rare forms of obesity. The program is designed for patients seeking education for themselves and their health care providers, assistance with insurance navigation at treatment initiation, injection support, and guidance on what to expect throughout treatment. For more information, contact patientsupport@rhythmtx.com.